Nature24d
Gene therapy articles from across Nature Portfolio
Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...
Gene therapy loses luster as investors eye quicker returns from weight-loss drugs
Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
New Scientist14d
What is CRISPR?
How CRISPR therapy could cure everything from cancer to infertility The imminent approval of the world's first CRISPR treatment for sickle cell disease is just the start: soon this gene-editing ...
University of California1d
Breathing new life: Working toward an inhalable gene therapy for cystic fibrosis
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
GEN - Genetic Engineering and Biotechnology News2d
Gene Therapy Tested in Mice Offers New Hope for People with Dravet Syndrome
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
The American Journal of Managed Care3d
Shaping Gene Therapy Approaches in Neuromuscular Disease
Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...